Zorevunersen (STK-001) - (EMPEROR)

October 5, 2026: Stoke Therapeutics (NASDAQ: STOK) said it completed an FDA meeting on its planned U.S. NDA for zorevunersen in Dravet syndrome, aligning on key submission elements including analysis of the Phase 3 EMPEROR study’s cognition/behavior endpoint. For the U.S. NDA, the Vineland-3 key secondary endpoint will be analyzed with a multicomponent MMRM assessment of four prespecified subdomains: expressive communication, receptive communication, interpersonal relationships, and personal skills.

EMPEROR is a global, double-blind, sham-controlled Phase 3 trial of intrathecal zorevunersen versus sham in children ages 2 to under 18 with Dravet syndrome and a qualifying SCN1A variant. The primary endpoint is change from baseline in log-transformed major motor seizure frequency at Week 28; key secondary endpoints are durability of seizure effect at Week 52 and adaptive functioning at Week 52. Additional endpoints include safety, CGI-C, CaGI-C, BSID-IV, and EQ-VAS.

Stoke said it has 5 years of safety and efficacy data from Phase 1/2a and the ongoing OLE. Phase 3 readout is anticipated in Q3 2027, with data submissions to FDA in 1H 2027 and planned NDA completion in 2H 2027, targeting potential U.S. approval and launch in early 2028.